Sandra Horning, MD, Roches chief medical officer and head of global product development, said, This FDA decision means people living with Erdheim-Chester disease
will now, for the first time, have an FDA-approved treatment option.
The US Food and Drug Administration has approved Zelboraf (vemurafenib) for Erdheim-Chester disease
with BRAF V600 mutation.
Swiss healthcare company Hoffman-LaRoche Inc reported on Monday the receipt of expanded approval from the US Food and Drug Administration for Zelboraf (vemurafenib) to include the treatment of certain adult patients with Erdheim-Chester Disease
(ECD) of the blood.
is a rare non-Langerhans histiocytosis, characterized by accumulation or proliferation of foamy histiocytes within long bones in nearly all cases.
Consensus guidelines for the diagnosis and clinical management of Erdheim-Chester disease
of the breast: a case report and review of the literature.
Variability in the efficacy of interferon-a in Erdheim-Chester disease
by patient and site of involvement: results in eight patients," Arthritis and Rheumatism, vol.
is a systemic histiocytic disorder diagnosed on the basis of a characteristic pattern of symmetric intramedullary sclerosis of long bones.
These include Simon Dedrick, who suffers from an extremely rare disorder called Erdheim-Chester disease
, of which there has been only 300 cases reported in the world.
The product is intended for Erdheim-Chester disease
(ECD) with BRAF V600 mutation, a rare, serious blood disease characterised by the abnormal multiplication of certain white blood cells called histiocytes.
They compared and contrasted the clinical, radiologic, and histologic characteristics of various causes of advanced fibrotic ILD, including connective tissue disease-associated ILD, chronic hypersensitivity pneumonitis, advanced pulmonary Langerhans cell histiocytosis, end-stage pulmonary sarcoidosis, Erdheim-Chester disease
, and Hermansky-Pudlak syndrome.
V600E] mutation, there is potential for vemurafenib, a BRAF enzyme inhibitor, as a targeted therapy as it was shown to exhibit dramatic effects in other dendritic and histiocytic neoplasms such as Erdheim-Chester disease
and Langerhans cell histiocytosis.